Ignite Creation Date:
2025-12-24 @ 11:53 PM
Ignite Modification Date:
2025-12-25 @ 9:49 PM
Study NCT ID:
NCT04806451
Status:
ACTIVE_NOT_RECRUITING
Last Update Posted:
2025-02-05
First Post:
2021-03-16
Is NOT Gene Therapy:
False
Has Adverse Events:
True
Brief Title:
Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)
Sponsor:
Organization:
Raw JSON
{'hasResults': True, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D000312', 'term': 'Adrenal Hyperplasia, Congenital'}, {'id': 'C535979', 'term': 'Congenital adrenal hyperplasia due to 21 hydroxylase deficiency'}], 'ancestors': [{'id': 'D047808', 'term': 'Adrenogenital Syndrome'}, {'id': 'D012734', 'term': 'Disorders of Sex Development'}, {'id': 'D014564', 'term': 'Urogenital Abnormalities'}, {'id': 'D052776', 'term': 'Female Urogenital Diseases'}, {'id': 'D005261', 'term': 'Female Urogenital Diseases and Pregnancy Complications'}, {'id': 'D000091642', 'term': 'Urogenital Diseases'}, {'id': 'D052801', 'term': 'Male Urogenital Diseases'}, {'id': 'D000013', 'term': 'Congenital Abnormalities'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D043202', 'term': 'Steroid Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D000307', 'term': 'Adrenal Gland Diseases'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}, {'id': 'D006058', 'term': 'Gonadal Disorders'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C000723083', 'term': 'crinecerfont'}]}}, 'resultsSection': {'moreInfoModule': {'pointOfContact': {'email': 'medinfo@neurocrine.com', 'phone': '877-641-3461', 'title': 'Neurocrine Medical Information Call Center', 'organization': 'Neurocrine Biosciences'}, 'certainAgreement': {'piSponsorEmployee': False, 'restrictiveAgreement': True}}, 'adverseEventsModule': {'timeFrame': 'Baseline up to Week 28', 'description': 'Per planned analysis all-cause mortality data were collected and reported for all randomized participants. Adverse events (serious and other) were collected and reported for all randomized participants who received at least 1 dose of study drug. Only DB period was complete at the time of data cutoff date.', 'eventGroups': [{'id': 'EG000', 'title': 'DB Period: Placebo', 'description': 'Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period.', 'otherNumAtRisk': 33, 'deathsNumAtRisk': 34, 'otherNumAffected': 22, 'seriousNumAtRisk': 33, 'deathsNumAffected': 0, 'seriousNumAffected': 4}, {'id': 'EG001', 'title': 'DB Period: Crinecerfont', 'description': "Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1.", 'otherNumAtRisk': 69, 'deathsNumAtRisk': 69, 'otherNumAffected': 51, 'seriousNumAtRisk': 69, 'deathsNumAffected': 0, 'seriousNumAffected': 1}], 'otherEvents': [{'term': 'Headache', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 17}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 15}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 8}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 10}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Upper respiratory tract infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 8}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Nasopharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 6}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 7}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Influenza', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 6}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Abdominal pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 5}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Coronavirus infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 5}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Fatigue', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 5}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Nasal congestion', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 5}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Cough', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 4}], 'organSystem': 'Respiratory, thoracic and mediastinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Dizziness', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 4}], 'organSystem': 'Nervous system disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Nausea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 4}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Pharyngitis streptococcal', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Viral infection', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 4}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': '17-hydroxyprogesterone increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Blood androstenedione increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 3}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Blood corticotrophin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 2}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Diarrhoea', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 3}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 2}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Blood insulin increased', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 1}], 'organSystem': 'Investigations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Constipation', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 1}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Tonsillitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}], 'seriousEvents': [{'term': 'Pyrexia', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 0}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 1}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Vomiting', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 2}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Gastrointestinal disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Pharyngitis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Chest pain', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'General disorders', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Gastroenteritis norovirus', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Gastroenteritis', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}, {'term': 'Gastroenteritis viral', 'stats': [{'groupId': 'EG000', 'numAtRisk': 33, 'numAffected': 1}, {'groupId': 'EG001', 'numAtRisk': 69, 'numAffected': 0}], 'organSystem': 'Infections and infestations', 'assessmentType': 'SYSTEMATIC_ASSESSMENT', 'sourceVocabulary': 'MedDRA 26.0'}], 'frequencyThreshold': '5'}, 'outcomeMeasuresModule': {'outcomeMeasures': [{'type': 'PRIMARY', 'title': 'Change From Baseline in Serum Androstenedione at Week 4', 'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'OG000'}, {'value': '69', 'groupId': 'OG001'}]}], 'groups': [{'id': 'OG000', 'title': 'Placebo', 'description': 'Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.'}, {'id': 'OG001', 'title': 'Crinecerfont', 'description': "Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight."}], 'classes': [{'categories': [{'measurements': [{'value': '70.986', 'spread': '56.198', 'groupId': 'OG000'}, {'value': '-196.789', 'spread': '39.369', 'groupId': 'OG001'}]}]}], 'analyses': [{'pValue': '0.0002', 'groupIds': ['OG000', 'OG001'], 'paramType': 'LS Mean Difference', 'ciNumSides': 'TWO_SIDED', 'ciPctValue': '95', 'paramValue': '-267.775', 'ciLowerLimit': '-403.427', 'ciUpperLimit': '-132.124', 'dispersionType': 'STANDARD_ERROR_OF_MEAN', 'dispersionValue': '68.313', 'statisticalMethod': 'ANCOVA', 'nonInferiorityType': 'SUPERIORITY'}], 'paramType': 'LEAST_SQUARES_MEAN', 'timeFrame': 'Baseline, Week 4', 'description': 'Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.', 'unitOfMeasure': 'ng/dL', 'dispersionType': 'Standard Error', 'reportingStatus': 'POSTED', 'populationDescription': 'Full Analysis Set included all randomized participants.'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4', 'timeFrame': 'Baseline, Week 4', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28', 'timeFrame': 'Baseline, Week 28', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28', 'timeFrame': 'Week 28', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28', 'timeFrame': 'Baseline, Week 28', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28', 'timeFrame': 'Baseline, Week 28', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}, {'type': 'SECONDARY', 'title': 'Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28', 'timeFrame': 'Baseline, Week 28', 'reportingStatus': 'NOT_POSTED', 'denomUnitsSelected': 'Participants'}]}, 'participantFlowModule': {'groups': [{'id': 'FG000', 'title': 'Placebo', 'description': 'Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.'}, {'id': 'FG001', 'title': 'Crinecerfont', 'description': "Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight."}], 'periods': [{'title': 'DB Treatment Period (28 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '34'}, {'groupId': 'FG001', 'numSubjects': '69'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '33'}, {'groupId': 'FG001', 'numSubjects': '69'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}, {'groupId': 'FG001', 'numSubjects': '66'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '3'}]}], 'dropWithdraws': [{'type': 'Withdrawal by Subject', 'reasons': [{'groupId': 'FG000', 'numSubjects': '3'}, {'groupId': 'FG001', 'numSubjects': '1'}]}, {'type': 'Adverse Event', 'reasons': [{'groupId': 'FG000', 'numSubjects': '0'}, {'groupId': 'FG001', 'numSubjects': '2'}]}]}, {'title': 'OL Treatment Period (24 Weeks)', 'milestones': [{'type': 'STARTED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}, {'groupId': 'FG001', 'numSubjects': '66'}]}, {'type': 'Received at Least 1 Dose of Study Drug', 'achievements': [{'groupId': 'FG000', 'numSubjects': '31'}, {'groupId': 'FG001', 'numSubjects': '66'}]}, {'type': 'COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '15'}, {'groupId': 'FG001', 'numSubjects': '29'}]}, {'type': 'NOT COMPLETED', 'achievements': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '37'}]}], 'dropWithdraws': [{'type': 'Ongoing in the Study', 'reasons': [{'groupId': 'FG000', 'numSubjects': '16'}, {'groupId': 'FG001', 'numSubjects': '37'}]}]}], 'preAssignmentDetails': 'The study includes a double-blind (DB) placebo-controlled treatment period, an open-label (OL) treatment period, and an open-label extension (OLE) period. The OL treatment period and OLE period of the study is ongoing. Only the primary analysis results (as of 09 August 2023 data cutoff date) have been reported. The final results will be reported after completion of the study.'}, 'baselineCharacteristicsModule': {'denoms': [{'units': 'Participants', 'counts': [{'value': '34', 'groupId': 'BG000'}, {'value': '69', 'groupId': 'BG001'}, {'value': '103', 'groupId': 'BG002'}]}], 'groups': [{'id': 'BG000', 'title': 'Placebo', 'description': 'Participants received crinecerfont-matched placebo orally twice daily for 28 weeks during the DB placebo-controlled treatment period. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight.'}, {'id': 'BG001', 'title': 'Crinecerfont', 'description': "Participants received crinecerfont orally twice daily for 28 weeks during the DB placebo-controlled treatment period. Dose assignment from Day 1 to Week 28 was based on the participant's weight at Day 1. After the 28-week DB placebo-controlled treatment period, there was a 24-week, OL treatment period, during which all participants received crinecerfont at doses based on their Week 28 body weight."}, {'id': 'BG002', 'title': 'Total', 'description': 'Total of all reporting groups'}], 'measures': [{'title': 'Age, Continuous', 'classes': [{'categories': [{'measurements': [{'value': '12.142', 'spread': '3.690', 'groupId': 'BG000'}, {'value': '12.022', 'spread': '3.405', 'groupId': 'BG001'}, {'value': '12.061', 'spread': '3.484', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'years', 'dispersionType': 'STANDARD_DEVIATION'}, {'title': 'Sex: Female, Male', 'classes': [{'categories': [{'title': 'Female', 'measurements': [{'value': '16', 'groupId': 'BG000'}, {'value': '34', 'groupId': 'BG001'}, {'value': '50', 'groupId': 'BG002'}]}, {'title': 'Male', 'measurements': [{'value': '18', 'groupId': 'BG000'}, {'value': '35', 'groupId': 'BG001'}, {'value': '53', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Ethnicity (NIH/OMB)', 'classes': [{'categories': [{'title': 'Hispanic or Latino', 'measurements': [{'value': '3', 'groupId': 'BG000'}, {'value': '8', 'groupId': 'BG001'}, {'value': '11', 'groupId': 'BG002'}]}, {'title': 'Not Hispanic or Latino', 'measurements': [{'value': '25', 'groupId': 'BG000'}, {'value': '52', 'groupId': 'BG001'}, {'value': '77', 'groupId': 'BG002'}]}, {'title': 'Unknown or Not Reported', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Race/Ethnicity, Customized', 'classes': [{'title': 'Race', 'categories': [{'title': 'American Indian or Alaska Native', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Asian', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '7', 'groupId': 'BG001'}, {'value': '9', 'groupId': 'BG002'}]}, {'title': 'Black or African American', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '3', 'groupId': 'BG002'}]}, {'title': 'White', 'measurements': [{'value': '23', 'groupId': 'BG000'}, {'value': '42', 'groupId': 'BG001'}, {'value': '65', 'groupId': 'BG002'}]}, {'title': 'Native Hawaiian or Other Pacific Islander', 'measurements': [{'value': '0', 'groupId': 'BG000'}, {'value': '1', 'groupId': 'BG001'}, {'value': '1', 'groupId': 'BG002'}]}, {'title': 'Other', 'measurements': [{'value': '2', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '5', 'groupId': 'BG002'}]}, {'title': 'Multiple', 'measurements': [{'value': '1', 'groupId': 'BG000'}, {'value': '3', 'groupId': 'BG001'}, {'value': '4', 'groupId': 'BG002'}]}, {'title': 'Not Collected', 'measurements': [{'value': '6', 'groupId': 'BG000'}, {'value': '9', 'groupId': 'BG001'}, {'value': '15', 'groupId': 'BG002'}]}]}], 'paramType': 'COUNT_OF_PARTICIPANTS', 'unitOfMeasure': 'Participants'}, {'title': 'Serum Androstenedione Concentration', 'classes': [{'categories': [{'measurements': [{'value': '483.32', 'spread': '455.64', 'groupId': 'BG000'}, {'value': '404.96', 'spread': '464.10', 'groupId': 'BG001'}, {'value': '430.83', 'spread': '460.58', 'groupId': 'BG002'}]}]}], 'paramType': 'MEAN', 'unitOfMeasure': 'nanograms (ng)/deciliter (dL)', 'dispersionType': 'STANDARD_DEVIATION'}], 'populationDescription': 'Full Analysis Set included all randomized participants.'}}, 'documentSection': {'largeDocumentModule': {'largeDocs': [{'date': '2022-05-10', 'size': 702609, 'label': 'Study Protocol', 'hasIcf': False, 'hasSap': False, 'filename': 'Prot_000.pdf', 'typeAbbrev': 'Prot', 'uploadDate': '2024-12-27T10:18', 'hasProtocol': True}, {'date': '2023-09-18', 'size': 648492, 'label': 'Statistical Analysis Plan', 'hasIcf': False, 'hasSap': True, 'filename': 'SAP_001.pdf', 'typeAbbrev': 'SAP', 'uploadDate': '2024-12-27T10:19', 'hasProtocol': False}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL'}, 'enrollmentInfo': {'type': 'ACTUAL', 'count': 103}}, 'statusModule': {'overallStatus': 'ACTIVE_NOT_RECRUITING', 'startDateStruct': {'date': '2021-06-25', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-01', 'dispFirstSubmitDate': '2024-03-05', 'completionDateStruct': {'date': '2027-08', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-01-10', 'studyFirstSubmitDate': '2021-03-16', 'resultsFirstSubmitDate': '2025-01-10', 'studyFirstSubmitQcDate': '2021-03-16', 'dispFirstPostDateStruct': {'date': '2024-03-07', 'type': 'ACTUAL'}, 'lastUpdatePostDateStruct': {'date': '2025-02-05', 'type': 'ACTUAL'}, 'resultsFirstSubmitQcDate': '2025-01-10', 'studyFirstPostDateStruct': {'date': '2021-03-19', 'type': 'ACTUAL'}, 'resultsFirstPostDateStruct': {'date': '2025-02-05', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2023-03-10', 'type': 'ACTUAL'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Change From Baseline in Serum Androstenedione at Week 4', 'timeFrame': 'Baseline, Week 4', 'description': 'Blood serum samples were collected for the analysis of serum androstenedione concentrations. Least square (LS) mean and standard error (SE) were calculated using analysis of covariance (ANCOVA) model.'}], 'secondaryOutcomes': [{'measure': 'Change From Baseline in Serum 17-hydroxyprogesterone (17-OHP) at Week 4', 'timeFrame': 'Baseline, Week 4'}, {'measure': 'Percent Change From Baseline in Glucocorticoid Daily Dose at Week 28', 'timeFrame': 'Baseline, Week 28'}, {'measure': 'Number of Participants Who Achieved a Reduction to Physiologic Glucocorticoid Dose While Maintaining Androstenedione Control at Week 28', 'timeFrame': 'Week 28'}, {'measure': 'Change From Baseline in Body Mass Index (BMI) Standard Deviation Score (SDS) at Week 28', 'timeFrame': 'Baseline, Week 28'}, {'measure': 'Change From Baseline in Mean 24-hour Salivary 17-OHP at Week 28', 'timeFrame': 'Baseline, Week 28'}, {'measure': 'Change From Baseline in the Ratio of Bone Age to Chronological Age (BA:CA) at Week 28', 'timeFrame': 'Baseline, Week 28'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['classic congenital adrenal hyperplasia (CAH)', '21-hydroxylase deficiency'], 'conditions': ['Congenital Adrenal Hyperplasia']}, 'referencesModule': {'references': [{'pmid': '38828945', 'type': 'DERIVED', 'citation': 'Sarafoglou K, Kim MS, Lodish M, Felner EI, Martinerie L, Nokoff NJ, Clemente M, Fechner PY, Vogiatzi MG, Speiser PW, Auchus RJ, Rosales GBG, Roberts E, Jeha GS, Farber RH, Chan JL; CAHtalyst Pediatric Trial Investigators. Phase 3 Trial of Crinecerfont in Pediatric Congenital Adrenal Hyperplasia. N Engl J Med. 2024 Aug 8;391(6):493-503. doi: 10.1056/NEJMoa2404655. Epub 2024 Jun 2.'}]}, 'descriptionModule': {'briefSummary': 'This is a Phase 3 study to evaluate the efficacy, safety, and tolerability of crinecerfont versus placebo administered for 28 weeks in approximately 81 pediatric participants with classic congenital adrenal hyperplasia (CAH) due to 21-hydroxylase deficiency. The study consists of a 28-week double blind, placebo-controlled period, followed by 24 weeks of open-label treatment with crinecerfont. Subsequently, participants may elect to participate in the open-label extension (OLE) period. The duration of participation in the study is approximately 14 months for the core study and will be a variable amount of time per participant for the OLE (estimated to be approximately 3 years).'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD'], 'maximumAge': '17 Years', 'minimumAge': '2 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Be willing and able to adhere to the study procedures, including all requirements at the study center, and return for the follow-up visit.\n* Have a medically confirmed diagnosis of classic CAH due to 21-hydroxylase deficiency.\n* Be on a stable steroid regimen.\n* Have elevated androgen levels.\n* Participants of childbearing potential must be abstinent or agree to use appropriate birth control during the study.\n\nExclusion Criteria:\n\n* Have a diagnosis of any of the other forms of classic CAH.\n* Have a history of bilateral adrenalectomy, hypopituitarism, or other condition requiring chronic glucocorticoid therapy.\n* Have a clinically significant unstable medical condition or chronic disease other than CAH.\n* Have a history of cancer unless considered to be cured.\n* Have a known history of clinically significant arrhythmia or abnormalities on electrocardiogram (ECG).\n* Have a known hypersensitivity to any corticotropin-releasing hormone antagonist.\n* Have received an investigational drug within 30 days before initial screening or plan to use an investigational drug (other than the study drug) during the study.\n* Have current substance dependence or substance (drug) or alcohol abuse.\n* Have had a significant blood loss or donated blood or blood products within 8 weeks prior to the study.'}, 'identificationModule': {'nctId': 'NCT04806451', 'briefTitle': 'Global Safety and Efficacy Registration Study of Crinecerfont in Pediatric Participants With Classic Congenital Adrenal Hyperplasia (CAHtalyst Pediatric Study)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Neurocrine Biosciences'}, 'officialTitle': 'A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Safety and Efficacy of Crinecerfont (NBI-74788) in Pediatric Subjects With Classic Congenital Adrenal Hyperplasia, Followed by Open-Label Treatment', 'orgStudyIdInfo': {'id': 'NBI-74788-CAH2006'}, 'secondaryIdInfos': [{'id': '2020-004381-19', 'type': 'EUDRACT_NUMBER'}, {'id': '2023-509170-33-00', 'type': 'CTIS'}]}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Crinecerfont', 'description': 'Crinecerfont solution or capsule, administered orally, twice daily for 28 weeks during the placebo-controlled treatment period, followed by active treatment with crinecerfont for at least 24 weeks.', 'interventionNames': ['Drug: Crinecerfont']}, {'type': 'PLACEBO_COMPARATOR', 'label': 'Placebo', 'description': 'Placebo solution or capsule, administered orally, twice daily for 28 weeks, followed by active treatment with crinecerfont for at least 24 weeks.', 'interventionNames': ['Drug: Crinecerfont', 'Drug: Placebo']}], 'interventions': [{'name': 'Crinecerfont', 'type': 'DRUG', 'otherNames': ['NBI-74788', 'Crenessity'], 'description': 'CRF type 1 receptor antagonist', 'armGroupLabels': ['Crinecerfont', 'Placebo']}, {'name': 'Placebo', 'type': 'DRUG', 'description': 'Non-active dosage form', 'armGroupLabels': ['Placebo']}]}, 'contactsLocationsModule': {'locations': [{'zip': '35233', 'city': 'Birmingham', 'state': 'Alabama', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 33.52066, 'lon': -86.80249}}, {'zip': '90027', 'city': 'Los Angeles', 'state': 'California', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 34.05223, 'lon': -118.24368}}, {'zip': '92868', 'city': 'Orange', 'state': 'California', 'country': 'United States', 'facility': 'Neurocrine Clinical Site', 'geoPoint': {'lat': 33.78779, 'lon': -117.85311}}, {'zip': '92123', 'city': 'San Diego', 'state': 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